Olezarsen (Tryngolza) Approved for Familial Chylomicronemia Syndrome
Summary
MHRA granted approval to olezarsen (Tryngolza) on 10 April 2026 to Swedish Orphan Biovitrum AB (publ) for treating adults with familial chylomicronemia syndrome (FCS), an inherited condition causing dangerously high triglyceride levels. A clinical study of 66 adults demonstrated that Tryngolza achieved an average 32% triglyceride reduction after 6 months versus a 12% average increase in the placebo group, with benefits sustained and improved after one year. Healthcare providers and patients in the UK now have a new treatment option for this rare, potentially life-threatening condition.
“This approval provides a new treatment option for adults living with familial chylomicronemia syndrome, an inherited condition that can lead to inflammation of the pancreas, causing severe pain and potentially life-threatening complications.”
About this source
The Medicines and Healthcare products Regulatory Agency is the UK's medicines and medical devices regulator. MHRA publications include drug safety alerts, device field safety notices, Class 1-4 defect recalls, guidance updates, and the monthly medicines shortage list. Around 55 publications a month. Device field safety notices in particular are useful because MHRA often publishes them hours before other European regulators (ANSM, BfArM) surface the same recall. Watch this if you manufacture or distribute medicines or medical devices in the UK and EU, run a hospital pharmacy, advise on MHRA licensing, or follow post-market surveillance signals across European markets.
What changed
MHRA has approved olezarsen (Tryngolza), an injectable antisense oligonucleotide therapy administered subcutaneously, for the treatment of familial chylomicronemia syndrome (FCS) in adults. The approval was granted via the International Recognition Procedure (IRP) Route B and is held by Swedish Orphan Biovitrum AB (publ). Clinical evidence from a 66-patient study demonstrated a 32% average triglyceride reduction at 6 months compared to placebo, with further improvement observed at one year and fewer acute pancreatitis cases reported.
Pharmaceutical companies and healthcare providers should note this approval as it expands treatment options for patients with this rare genetic disorder. Prescribers treating FCS patients should review the Summary of Product Characteristics and Patient Information Leaflets expected on the MHRA Products website within 7 days of approval. Ongoing safety monitoring will continue as the drug is used more widely.
Archived snapshot
Apr 12, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Press release
MHRA approves olezarsen (Tryngolza) for the treatment of familial chylomicronemia syndrome
MHRA approves olezarsen (Tryngolza) for the treatment of familial chylomicronemia syndrome
From: Medicines and Healthcare products Regulatory Agency Published 10 April 2026
The Medicines and Healthcare products Regulatory Agency (MHRA) has today 10 April 2026, approved olezarsen (Tryngolza) to help treat adults with familial chylomicronemia syndrome (FCS).
FCS is an inherited disease that gives rise to abnormally high levels of fats called triglycerides in the blood. This can lead to inflammation of the pancreas, causing severe pain, lasting damage to the pancreas, and can be life threatening.
Olezarsen is administered as an injection under the skin, usually into the stomach area, the front of the thighs, or the back of the upper arms.
In a main study involving 66 adults with FCS, olezarsen was shown to significantly reduce triglyceride levels in the blood. All patients followed a controlled diet and received either Tryngolza or a placebo.
After 6 months, patients treated with Tryngolza saw an average reduction in triglycerides of 32%, compared with an average increase of 12% in those given placebo. These benefits were maintained and further improved after one year, with fewer cases of acute pancreatitis reported in patients taking Tryngolza.
Julian Beach, Interim Executive Director of Healthcare Quality and Access at the MHRA, said:
“This approval provides a new treatment option for adults living with familial chylomicronemia syndrome, an inherited condition that can lead to inflammation of the pancreas, causing severe pain and potentially life-threatening complications.
“As with all medicines, we will continue to closely monitor the safety and effectiveness of olezarsen as it is used more widely.”
Notes to editors
- The approval was granted on 10/04/2026 to Swedish Orphan Biovitrum AB (publ)
- This product was submitted and approved via IRP Route B
- More information can be found in the Summary of Product Characteristics and Patient Information Leaflets which will be published on the MHRA Products website within 7 days of approval.
- The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
- The MHRA is an executive agency of the Department of Health and Social Care.
- For media enquiries, please contact newscentre@mhra.gov.uk or call 020 3080 7651.
Share this page
The following links open in a new tab
Updates to this page
Published 10 April 2026
Mentioned entities
Related changes
Get daily alerts for MHRA Guidance & Safety
Daily digest delivered to your inbox.
Free. Unsubscribe anytime.
Source
About this page
Every important government, regulator, and court update from around the world. One place. Real-time. Free. Our mission
Source document text, dates, docket IDs, and authority are extracted directly from MHRA.
The summary, classification, recommended actions, deadlines, and penalty information are AI-generated from the original text and may contain errors. Always verify against the source document.
Classification
Who this affects
Taxonomy
Browse Categories
Get alerts for this source
We'll email you when MHRA Guidance & Safety publishes new changes.
Subscribed!
Optional. Filters your digest to exactly the updates that matter to you.